Clinical Trials for Nervous System Disorders: Current Research and Treatment Options

There are currently 14 ongoing clinical trials investigating treatments for various nervous system disorders. These trials focus on conditions including multiple sclerosis, Parkinson’s disease, Alzheimer’s disease, spinocerebellar ataxia, and sleep-wake disorders. Research is being conducted across multiple European countries, testing both established medications in new contexts and novel investigational drugs to improve patient outcomes and quality of life.

Clinical trial locations

• Austria
  • Study on the Safety and Effects of NSC001 and Trospium Chloride for Patients with Mild to Moderate Alzheimer’s Disease
• Belgium
  • Study on Metformin for Delaying Progression in Non-Active Progressive Multiple Sclerosis Patients
• Bulgaria
  • Study Comparing Gadoquatrane with Gadoteric Acid, Gadobutrol, and Gadoteridol for MRI in Adults with Brain or Spinal Cord Issues
  • Study on the Safety and Effectiveness of Peginterferon Beta-1a for Children and Teens with Relapsing-Remitting Multiple Sclerosis
• Croatia
  • Study on the Safety and Effectiveness of Peginterferon Beta-1a for Children and Teens with Relapsing-Remitting Multiple Sclerosis
• Czechia
  • Continued Access Study for Patients with Neurological Disease Using Ocrelizumab and Drug Combination
  • Study Comparing Gadoquatrane with Gadoteric Acid, Gadobutrol, and Gadoteridol for MRI in Adults with Brain or Spinal Cord Issues
  • Study on the Safety and Effectiveness of Peginterferon Beta-1a for Children and Teens with Relapsing-Remitting Multiple Sclerosis
• Denmark
  • Study on the Safety and Effects of NPI-001 (Acetylcysteine Amide) for Patients Aged 50-85 with Mild Cognitive Impairment or Mild Alzheimer’s Disease
• France
  • Study Comparing Rituximab and Ocrelizumab for Patients with Relapsing-Remitting Multiple Sclerosis
  • Continued Access Study for Patients with Neurological Disease Using Ocrelizumab and Drug Combination
  • Safety Study of Tasimelteon for Treating Non-24-Hour Sleep-Wake Disorder in Blind Individuals Without Light Perception
  • Study Comparing Gadoquatrane with Gadoteric Acid, Gadobutrol, and Gadoteridol for MRI in Adults with Brain or Spinal Cord Issues
  • Study on the Physical Impact of Multiple Sclerosis and Continued Access to Ocrelizumab for Patients Previously in a Related Study
• Germany
  • Continued Access Study for Patients with Neurological Disease Using Ocrelizumab and Drug Combination
  • Study Comparing Gadoquatrane with Gadoteric Acid, Gadobutrol, and Gadoteridol for MRI in Adults with Brain or Spinal Cord Issues
  • Study on the Effects of Donepezil on Cognitive and Motor Functions in Elderly Patients with Neurological Disorders
  • Study on the Physical Impact of Multiple Sclerosis and Continued Access to Ocrelizumab for Patients Previously in a Related Study
  • Study on the Safety and Effects of NSC001 and Trospium Chloride for Patients with Mild to Moderate Alzheimer’s Disease
• Hungary
  • Study Comparing Gadoquatrane with Gadoteric Acid, Gadobutrol, and Gadoteridol for MRI in Adults with Brain or Spinal Cord Issues
  • Study on the Safety and Effectiveness of Peginterferon Beta-1a for Children and Teens with Relapsing-Remitting Multiple Sclerosis
• Iceland
  • Study on the Safety and Effects of NPI-001 (Acetylcysteine Amide) for Patients Aged 50-85 with Mild Cognitive Impairment or Mild Alzheimer’s Disease
• Italy
  • Safety Study of Afamelanotide for Patients with Early Parkinson’s Disease
  • Study Comparing Gadoquatrane with Gadoteric Acid, Gadobutrol, and Gadoteridol for MRI in Adults with Brain or Spinal Cord Issues
  • Study of Riluzole for Patients with Spinocerebellar Ataxia Type 7

Study Comparing Rituximab and Ocrelizumab for Patients with Relapsing-Remitting Multiple Sclerosis

This trial is comparing two medications for people with relapsing-remitting multiple sclerosis, a condition where the immune system attacks the protective covering of nerves. The study will help determine which medication works better at managing symptoms and preventing disease progression.

Inclusion criteria: Participants must be between 18 and 55 years old with confirmed relapsing-remitting MS. They need to have shown disease activity, such as at least one relapse or a new brain lesion on MRI, within the past 12 months. Their EDSS score, which measures disability, must be 5 or less. Women of childbearing potential must use effective birth control during the study and for 12 months after the last dose. All participants must have a recent brain MRI scan and social insurance coverage.

Exclusion criteria: People without a diagnosis of multiple sclerosis, those outside the specified age range, individuals unable to follow study procedures, those with recent infections or allergic reactions to study medications, and pregnant or breastfeeding women cannot participate. People currently in another clinical trial or using certain interfering medications are also excluded.

Study focus: The trial evaluates whether Rituximab and Ocrelizumab are equally effective at keeping patients free of disease activity over two years. Researchers will monitor relapse frequency, disability progression, quality of life, and safety throughout the study period.

Investigational drugs: Rituximab and Ocrelizumab are both monoclonal antibodies that target and reduce specific immune cells called B cells, which contribute to the disease. Both are given as infusions directly into the bloodstream.

Study of Riluzole for Patients with Spinocerebellar Ataxia Type 7

This study tests whether Riluzole can help stabilize symptoms in people with spinocerebellar ataxia type 7, a rare genetic disorder affecting movement and vision. The medication, already used for another neurological condition, may help protect nerve cells from damage.

Inclusion criteria: Participants must be over 6 years old with a positive genetic test for SCA7. Both males and females can participate. For minors, parents or legal representatives must provide informed consent.

Exclusion criteria: People with other serious health conditions that could interfere with the study, those who are pregnant or breastfeeding, individuals who participated in another trial within 30 days, those with drug or alcohol abuse history within the past year, people unable to comply with study procedures, those with known allergies to study medications, individuals with certain eye diseases, those who had major surgery within 3 months, and people with uncontrolled high blood pressure or severe liver or kidney disease cannot participate.

Study focus: The trial examines whether Riluzole can keep patients’ movement abilities and vision stable over 18 months. Researchers will use the SARA score to measure movement coordination and conduct vision tests to track any changes.

Investigational drugs: Riluzole, taken as 50 mg tablets by mouth, works by modulating certain neurotransmitters in the brain to help protect nerve cells. It is classified as a neuroprotective agent.

Study on the Effects of Donepezil on Cognitive and Motor Functions in Elderly Patients with Neurological Disorders

This trial investigates how a single dose of donepezil hydrochloride affects thinking and movement functions in healthy older adults. The medication increases levels of acetylcholine in the brain, which is important for memory and learning.

Inclusion criteria: Healthy volunteers aged 50 or older who are right-handed with a BMI between 18 and 32 kg/m² can participate. They must be suitable for MRI scans and provide written informed consent after understanding the study’s nature and procedures.

Exclusion criteria: People with neurological disorders, those outside the specified age range, and individuals from vulnerable populations cannot participate.

Study focus: The study measures how donepezil affects participants’ ability to perform tasks requiring both thinking and physical movement. Brain activity is monitored using MRI during these tasks to understand the medication’s effects on cognitive and motor functions.

Investigational drugs: Donepezil hydrochloride, a 5 mg oral tablet, is a cholinesterase inhibitor that prevents the breakdown of acetylcholine, thereby enhancing nerve cell communication.

Study on the Physical Impact of Multiple Sclerosis and Continued Access to Ocrelizumab for Patients Previously in a Related Study

This extension study ensures that patients who previously participated in related research can continue receiving Ocrelizumab treatment if they don’t have local access to it. The study also evaluates the physical impact of multiple sclerosis from the patient’s perspective.

Inclusion criteria: Participants must have signed the extension study consent form and been receiving Ocrelizumab in a previous study without local access to the medication. The first dose in this extension must be received no earlier than 5 months after the last treatment. Women of childbearing potential need a negative pregnancy test within 24 hours before the first dose and must use acceptable birth control methods.

Exclusion criteria: People without a multiple sclerosis diagnosis, those outside the specified age range, and individuals from vulnerable populations cannot participate.

Study focus: The trial monitors how patients perceive the physical impact of multiple sclerosis while continuing Ocrelizumab treatment for up to five years. Researchers track changes in physical function and record any side effects or adverse events.

Investigational drugs: Ocrelizumab is a monoclonal antibody given as either a 300 mg infusion into a vein or a 920 mg injection under the skin. It targets CD20-positive B cells, which play a role in the abnormal immune response damaging the nervous system.

Study on the Safety and Effects of NPI-001 (Acetylcysteine Amide) for Patients Aged 50-85 with Mild Cognitive Impairment or Mild Alzheimer’s Disease

This trial tests a new treatment called NPI-001 to see if it is safe and well-tolerated by people with mild cognitive impairment or mild dementia due to Alzheimer’s disease. The study also examines whether the medication affects harmful substances in the brain associated with the condition.

Inclusion criteria: Participants must be between 50 and 84 years old with mild cognitive impairment or mild Alzheimer’s dementia, receiving standard care. They need an MMSE score between 21 and 27 and must be willing to undergo blood tests and brain scans. A spouse, close relative, or caregiver must serve as a study partner. If taking other medications, doses must have been stable for at least 3 months. Participants must be able to read, write, and speak clearly for cognitive tests.

Exclusion criteria: People without an Alzheimer’s disease diagnosis, those outside the age range, individuals from vulnerable populations, those with medical conditions making participation unsafe, people currently in another trial, individuals with certain brain conditions interfering with results, those unable to undergo MRI, and people with uncontrolled health conditions cannot participate.

Study focus: The study assesses safety and tolerability of NPI-001 over 12 months while monitoring changes in brain markers using MRI scans and blood tests. Researchers also evaluate cognitive function and any side effects experienced by participants.

Investigational drugs: NPI-001 (AT-001) is an oral tablet being studied for its potential to help manage symptoms of Alzheimer’s disease by targeting specific pathways in the brain thought to be involved in disease progression.

Study on the Safety and Effects of NSC001 and Trospium Chloride for Patients with Mild to Moderate Alzheimer’s Disease

This study examines whether NSC001, a medication targeting specific brain receptors, can improve symptoms in people with mild to moderate Alzheimer’s disease. The trial also tests whether combining NSC001 with Trospium makes treatment more effective.

Inclusion criteria: Participants and a reliable caregiver must provide informed consent. Participants need an MMSE score between 18 and 26 and a Modified Hachinski Ischemic Scale score of 4 or less. They must have been on stable acetylcholinesterase inhibitor medications for at least three months.

Exclusion criteria: People with other serious medical conditions, those with severe allergic reaction history, individuals currently in another trial, those with significant psychiatric disorders or substance abuse issues, pregnant or breastfeeding women, people with uncontrolled medical conditions, those with known sensitivity to study medications, and individuals deemed unsuitable by the study doctor cannot participate.

Study focus: The trial evaluates safety and tolerability of NSC001 over 16 weeks, with or without Trospium. Researchers monitor cognitive function, vital signs, and any side effects to determine treatment effectiveness.

Investigational drugs: NSC001 is a selective muscarinic M1 agonist taken orally as capsules, designed to enhance brain function by targeting memory and learning receptors. Trospium, typically used for bladder problems, is tested in combination with NSC001 to potentially enhance treatment effectiveness.

Continued Access Study for Patients with Neurological Disease Using Ocrelizumab and Drug Combination

This extension study provides continued treatment access for patients with neurological disease who participated in previous research and lack local medication access. The study ensures patients can maintain their health and manage their condition effectively.

Inclusion criteria: Participants must sign the extension study consent form and be eligible to continue the treatment based on previous study requirements. The first dose must be received within the treatment window allowed by the previous study. Women of childbearing potential need a negative pregnancy test within 24 hours before the first dose.

Exclusion criteria: People without a neurological disease diagnosis, those not receiving treatment in the parent study, individuals with reasonable local access to treatment, those outside specified age ranges, people not part of specified clinical trial groups, and individuals not classified as vulnerable populations cannot participate.

Study focus: The trial provides ongoing treatment for patients who previously benefited from the therapy, ensuring seamless transition and continued care through July 31, 2029.

Investigational drugs: The study uses Ocrelizumab and supporting medications including Lidocaine Hydrochloride, Methylprednisolone Acetate, and Dexamethasone Acetate, administered through various routes to manage neurological conditions effectively.

Safety Study of Afamelanotide for Patients with Early Parkinson’s Disease

This trial evaluates the safety of afamelanotide in people with early Parkinson’s disease. The medication is a hormone analogue that may help protect and repair cells affected by the condition.

Inclusion criteria: Participants must be between 40 and 85 years old with a Parkinson’s disease diagnosis less than three years old. They should not yet require dopaminergic medication or be expected to need it within three months of enrollment. Both males and females can participate.

Exclusion criteria: People without a Parkinson’s disease diagnosis, those outside the specified age range, and individuals from vulnerable populations cannot participate.

Study focus: The study monitors safety over approximately nine months, assessing changes in protein levels, inflammation markers, brain imaging results, and cognitive function to understand how the treatment affects patients with early Parkinson’s disease.

Investigational drugs: Afamelanotide is administered as a subcutaneous injection under the skin. It works by stimulating melanin production, which may have neuroprotective effects, and is classified as a melanocortin receptor agonist.

Safety Study of Tasimelteon for Treating Non-24-Hour Sleep-Wake Disorder in Blind Individuals Without Light Perception

This study examines the safety of tasimelteon for treating sleep-wake cycle problems in blind individuals who cannot perceive light. The disorder makes it difficult for people to synchronize their internal body clock with the 24-hour day.

Inclusion criteria: Participants must be at least 18 years old with a BMI between 18 and 40 kg/m². They must have no light perception and a history of sleep problems within the past 3 months. Women of childbearing potential must use acceptable birth control for 35 days before the first dose and have a negative pregnancy test at screening and baseline visits. Participants must be affiliated with a social security system.

Exclusion criteria: People without a Non-24 Hour Sleep-Wake Disorder diagnosis, those outside the specified age range, and individuals from vulnerable populations cannot participate.

Study focus: The trial assesses the safety of tasimelteon over one year, monitoring side effects, mood changes, vital signs, and sleep patterns to determine how the medication affects sleep-wake cycles in blind individuals.

Investigational drugs: Tasimelteon is a 20 mg oral capsule taken daily. It works as a melatonin receptor agonist, mimicking the natural hormone involved in sleep regulation to help reset the body’s internal clock.

Study on the Safety and Effectiveness of TolDec and Immunotherapy for Patients with Relapsing-Remitting Multiple Sclerosis

This trial tests TolDec, a therapy using special cells taken from the patient’s own body and modified to help the immune system function better. The treatment is given alongside standard first-line immunotherapy for relapsing-remitting multiple sclerosis.

Inclusion criteria: Participants must be between 18 and 65 years old with RRMS diagnosed according to 2017 guidelines and disease duration less than 10 years. Their EDSS score must be between 0 and less than 5.5. They must be eligible for or already receiving first-line immunomodulatory treatment. Participants need to have experienced one or more relapses in the past 2 years or show new brain lesions on MRI. Women of childbearing potential must use highly effective birth control methods during the study.

Exclusion criteria: People without a multiple sclerosis diagnosis, those not receiving first-line immunotherapy for RRMS, individuals outside the specified age range, those not part of the specified clinical trial group, and people not from vulnerable populations cannot participate.

Study focus: The study evaluates whether TolDec combined with first-line immunotherapy is safe and effective at reducing new brain lesions and managing symptoms over 24 weeks, monitoring both efficacy and any adverse events.

Investigational drugs: TolDec uses autologous tolerogenic dendritic cells given as an intravenous injection to help the immune system better manage multiple sclerosis. First-line immunotherapy medications modify the immune system’s response to reduce inflammation and prevent further nervous system damage.

Summary

The 14 ongoing clinical trials for nervous system disorders reflect diverse research efforts across multiple European countries. Multiple sclerosis research dominates the landscape with six dedicated trials, examining both established treatments like Ocrelizumab and Rituximab as well as novel approaches like TolDec therapy. Several trials focus specifically on pediatric and extension populations, ensuring continued access to treatment for vulnerable groups.

Alzheimer’s disease research is also prominent, with three trials testing different investigational compounds including NPI-001 and NSC001, targeting various aspects of cognitive decline. These studies focus on early to moderate stages of the disease, reflecting the field’s emphasis on early intervention strategies.

Geographically, France and Germany lead with the highest number of trial locations, followed by Italy, Czechia, and Bulgaria. This concentration suggests these countries have established research infrastructure for neurological conditions. Several trials are multicountry studies, particularly those examining imaging agents and established medications, which helps ensure diverse patient populations and more generalizable results.

Notable trends include the repurposing of existing medications like metformin for new indications, the development of specialized pediatric formulations for conditions traditionally studied in adults, and continued focus on personalized immunotherapy approaches. Extension studies ensure that patients who respond well to experimental treatments can continue receiving them, addressing both ethical concerns and providing long-term safety data.